更新于：2024-11-01

Betibeglogene autotemcel

更新于：2024-11-01

概要

研发状态

概要

基本信息

药物类型

基因疗法、造血干细胞疗法

别名

2009 EGT-produced vector、Autologous CD34+ cell enriched population that contains hematopoietic stem cells transduced with lentiglobin BB305 lentiviral vector encoding the beta-A-T87Q-globin gene、Autologous CD34+ cells encoding βA-T87Q-globin gene

+ [11]

靶点

β-globin

作用机制

β-globin刺激剂(血红蛋白β链刺激剂)、基因转移

治疗领域

遗传病与畸形血液及淋巴系统疾病其他疾病

在研适应症

地中海贫血β地中海贫血镰状细胞血症+ [1]

非在研适应症

输血依赖性地中海贫血

原研机构

bluebird bio, Inc.

在研机构

bluebird bio, Inc.

非在研机构-

最高研发阶段批准上市

首次获批日期

欧盟 (2019-05-29),

输血依赖性β地中海贫血

最高研发阶段(中国)-

特殊审评优先药物（PRIME） (欧盟)、加速批准 (欧盟)、加速审评 (欧盟)、罕见儿科疾病 (美国)、孤儿药 (欧盟)、快速通道 (美国)、突破性疗法 (美国)、优先审评 (美国)、再生医学先进疗法 (美国)

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关联

项与 Betibeglogene autotemcel 相关的临床试验

JPRN-jRCTc071190041 / Not yet recruiting临床2期IIT

A single-arm, open-label, phase 2 study of autologous CD34+ stem cells-selected transplantation for severe systemic sclerosis - A single-arm, open-label, phase 2 study of autologous CD34+ stem cells-selected transplantation for severe systemic sclerosis

开始日期2019-01-04

申办/合作机构-

EUCTR2017-001275-23-GB / Unknown status临床2期IIT

Efficacy and safety of a cryopreserved formulation of autologous CD34+ haematopoietic stem cells transduced ex vivo with EFS lentiviral vector encoding for human ADA gene in subjects with Severe Combined Immunodeficiency (SCID) due to Adenosine Deaminase Deficiency

开始日期2017-09-21

申办/合作机构

Great Ormond Street Hospital Children's Charity

NCT03207009 / Completed临床3期

A Phase 3 Single Arm Study Evaluating the Efficacy and Safety of Gene Therapy in Subjects With Transfusion-dependent β-Thalassemia by Transplantation of Autologous CD34+ Stem Cells Transduced Ex Vivo With a Lentiviral βA-T87Q-Globin Vector in Subjects ≤50 Years of Age

This is a single-arm, multi-site, single-dose, Phase 3 study in approximately 18 participants less than or equal to (<=) 50 years of age with transfusion-dependent β-thalassemia (TDT), who have a β0/β0, β0/IVS-I-110, or IVS-I-110/IVS-I-110 genotype. The study will evaluate the efficacy and safety of autologous hematopoietic stem cell transplantation (HSCT) using LentiGlobin BB305 Drug Product.

开始日期2017-06-08

申办/合作机构

bluebird bio, Inc.

100 项与 Betibeglogene autotemcel 相关的临床结果

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100 项与 Betibeglogene autotemcel 相关的转化医学

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100 项与 Betibeglogene autotemcel 相关的专利（医药）

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项与 Betibeglogene autotemcel 相关的文献（医药）

2024-05-01·Blood Reviews

Revolutionising healing: Gene Editing's breakthrough against sickle cell disease

Review

作者: Strouboulis, John ; Dimitrievska, Marija ; Nicolaides, Kypros H ; Jacków-Malinowska, Joanna ; Shangaris, Panicos ; Miccio, Annarita ; El Hoss, Sara ; Bansal, Dravie ; Vitale, Marta

Recent advancements in gene editing illuminate new potential therapeutic approaches for Sickle Cell Disease (SCD), a debilitating monogenic disorder caused by a point mutation in the β-globin gene. Despite the availability of several FDA-approved medications for symptomatic relief, allogeneic hematopoietic stem cell transplantation (HSCT) remains the sole curative option, underscoring a persistent need for novel treatments. This review delves into the growing field of gene editing, particularly the extensive research focused on curing haemoglobinopathies like SCD. We examine the use of techniques such as CRISPR-Cas9 and homology-directed repair, base editing, and prime editing to either correct the pathogenic variant into a non-pathogenic or wild-type one or augment fetal haemoglobin (HbF) production. The article elucidates ways to optimize these tools for efficacious gene editing with minimal off-target effects and offers insights into their effective delivery into cells. Furthermore, we explore clinical trials involving alternative SCD treatment strategies, such as LentiGlobin therapy and autologous HSCT, distilling the current findings. This review consolidates vital information for the clinical translation of gene editing for SCD, providing strategic insights for investigators eager to further the development of gene editing for SCD.

2024-05-01·Current Opinion in Ophthalmology

Systemic medications for sickle cell disease and potential applications for sickle cell retinopathy

Review

作者: Garg, Anupam K ; Scott, Adrienne W

Purpose of reviewTo review the literature evaluating systemic medications for treatment of sickle cell disease (SCD) and their applications for sickle cell retinopathy.Recent FindingsPrior studies have demonstrated the efficacy of traditional systemic therapies in reducing the risk of development of sickle cell retinopathy. Since 2017, several new and promising disease-modifying therapies for sickle cell disease have been approved for clinical use, including the first genetic therapies such as exagamglogene autotemcel (exa-cel) and lovotibeglogene autotemcel (lovo-cel). These treatments have shown promising results for systemic management but are not widely utilized due to limited access and high cost. The efficacy of these therapies for the prevention of sickle cell retinopathy remains unknown and opens the door to new avenues for research. Furthermore, the role of systemic therapy for the management of hemoglobin SC (HbSC) disease, which has milder systemic effects but higher likelihood of causing retinopathy, remains poorly understood.SummaryHydroxyurea has been a mainstay of systemic management of SCD with prior work suggesting its ability to reduce the likelihood of developing retinopathy. There are several new and potentially curative systemic therapies for SCD, though their role in retinopathy prevention and management has not been studied extensively. Future studies are necessary to understand the implications of these emerging therapies for sickle cell retinopathy.

2024-04-01·International Immunopharmacology

Biomimetic nanoparticles for effective Celastrol delivery to targeted treatment of rheumatoid arthritis through the ROS-NF-κB inflammasome axis

Article

作者: Wang, Bo ; Wang, Xinggao ; Shen, Jiquan ; Hou, Ruixing

Previous study has indicated that Celastrol (Cel) has various physiological and pharmacological effects, including antibacterial, antioxidant, pro-apoptotic, anticancer and anti-rheumatoid arthritis (RA) effects. However, low water solubility, low oral bioavailability, narrow treatment window, and high incidence of systemic adverse reactions still limit the further clinical application of Cel. Here, aiming at effectively overcome those shortcomings of Cel to boost its beneficial effects for treating RA, we developed the leukosome (LEUKO) coated biomimetic nanoparticles (NPs) for the targeted delivery of Cel to arthritis injury area in RA. LEUKO were synthesized using membrane proteins purified from activated J774 macrophage. LEUKO and Cel-loaded LEUKO (Cel@LEUKO) were characterized using dynamic light scattering and transmission electron microscopy. Our results demonstrated that Cel@LEUKO can inhibit the inflammatory response of lipopolysaccharide (LPS) induced mouse monocyte macrophage leukemia cells (RAW264.7 cells) and human rheumatoid arthritis synovial fibroblasts (MH7A) cells through the inhibition of reactive oxygen species (ROS)-NF-κB pathway. In addition, research has shown that LEUKO effectively targets and transports Cel to the inflammatory site of RA, increased drug concentration in affected areas, reduced systemic toxicity of Cel, and reduced clinical symptoms, inflammatory infiltration, bone erosion, and serum inflammatory factors in collagen-induced arthritis (CIA) rats.

547

项与 Betibeglogene autotemcel 相关的新闻（医药）

2024-10-28

·EndPoints

Bipartisan letter questions FDA's 'narrow' interpretation for priority review vouchers

Sen. Tim Scott (R-SC) and Rep. Danny Davis (D-IL) are questioning the FDA’s “narrow” interpretation of “active ingredient” when it comes to awarding the potentially lucrative priority review vouchers. The letter , dated last Thursday and addressed to FDA Commissioner Rob Califf, focuses on the two FDA approvals of sickle cell gene therapies in late 2023. It explains how FDA’s recent administration of the PRV program — granting a PRV to one of the sickle cell gene therapies but not the other — “may not be fulfilling the program’s promise, original vision, and intent.” Scott and Davis question FDA on why it “made an initial decision to narrowly interpret the definition of ‘active ingredient’ in the PRV statute, resulting in the unexpected denial of at least one pediatric PRV.” But they also note that FDA “may be in the process of reconsidering its interpretation” so they want to make their thoughts clear. Bluebird bio had initially expected to win a PRV for its sickle cell gene therapy, approved in December 2023, and Novartis even drew up plans to purchase the PRV for $103 million. Ultimately, the FDA did not grant the PRV to bluebird, but Vertex Pharmaceuticals won a PRV for its gene therapy for sickle cell, approved on the same day as bluebird’s. A spokesperson told Endpoints News, “We believe that both the science and the congressional intent of the Rare Pediatric Disease Priority Review Voucher Program clearly support the issuance of a PRV associated with the approval of LYFGENIA and have continued to pursue a resolution with the FDA.” The FDA previously said that Lyfgenia contains the same active ingredient as bluebird’s gene therapy Zynteglo, approved for beta thalassemia. Scott and Davis explain how Congress did not define the term “active ingredient” in the law governing PRVs, but sought to prevent potential manufacturers from receiving PRVs for “simply making small tweaks to old drugs.” “The development of these complex gene therapies to treat an entirely new disease affecting thousands of patients can hardly be considered a ‘small tweak’ to an ‘old drug,'” the letter says, noting that Congress did not specify how FDA should determine if two active ingredients in an ex vivo gene therapy are the same or different. Scott and Davis add that FDA’s decision to interpret the law “in a narrower way than the statute requires – and one that flies directly against Congressional intent” risks disrupting “this delicate ecosystem, jeopardizing not only the availability of SCD treatments today, but also investments in complex and life-saving innovation for the future.” Editor’s note: Article updated with bluebird bio comment.

优先审批基因疗法并购

2024-10-24

·佰傲谷BioValley

兜售唯一临床管线，张锋公司的再次转向

日前，Editas Medicine发布了一则战略更新，将重心再次转向体内CRISPR基因编辑。同时，寻求其目前唯一的临床管线reni-cel（之前名为EDIT-301）的对外许可或合作伙伴。在Editas过去的研发中，其实可以看到其重心在体内或体外疗法的不断摇摆，小编研究了一下，发现还是蛮有意思的——从AAV体内-到HSPC体外-再到LNP体内。被兜售的reni-cel reni-cel是一款用于治疗镰状细胞病（SCD）和β-地中海贫血的CRISPR-Cas12a基因编辑药物。EDIT-301由来自患者的CD34+造血干细胞和祖细胞（HSPC）构成，这些细胞经过CRISPR-Cas12a编辑γ珠蛋白基因（HBG1和HBG2）启动子，能够持续产生的胎儿血红蛋白（HbF）。 reni-cel是Editas公司管线内唯一一款临床管线，忍痛放弃reni-cel的原因可能是过长的开发时间、潜在的市场挑战、公司资金短缺等。根据报告，reni-cel尽管展示出积极的早期临床试验结果，所有镰状细胞贫血病（SCD）患者均未发生血管闭塞事件VOE；所有β地中海贫血患者的总血红蛋白均早期稳步升高，高于输血非依赖性阈值9g/dL。但是距离推进上市，还需要几年的时间。而在目前的SCD和β-地贫治疗市场上，包括美国FDA在内的几个监管机构已经批准了另一种CRISPR疗法Casgevy，另外还有蓝鸟的Lyfgenia（SCD）和Zynteglo（β-地贫）。值得注意的是，Casgevy的开发商Vertex与Editas达成了协议，Vertex获得了CRISPR-Cas9基因编辑技术的非独家授权，用于包括Casgevy在内的SCD和β-地贫体外基因编辑药物。为此，Editas将获得来自Vertex的5000万美元预付款+外加5000万美元的或有付款和年度许可费。不过，前些时候，Editas为了提前变现，将Vertex许诺的年度许可费交易给了DRI Healthcare，换取5700万美元的现金。延伸阅读：张锋公司现金流告急，提前变现许可权益几年后的未来，SCD和β-地贫的市场竞争可能更为激烈，reni-cel的竞争力很难讲。以及长达数年的CRISPR产品临床开发，对于公司资金压力也更大。 Editas的总裁兼首席执行官在一份声明中表示：“我们相信，对于患者和我们股东来说，最好的选择是寻求代替方案，例如全球合作伙伴或者对外许可，这件允许我们与另一方或由另一方进一步开发或最终商业化reni-cel，并将使Editas在2025年的支出大幅度减少。” 目前，Editas公司已聘请投资银行Moelis & Company来领导寻找reni-cel的合作伙伴或收购者的工作。不过，reni-cel的临床工作还未完全停摆。Editas报告将会对针对SCD的1/2/3期试验RUBY中和针对β-地贫的试验EdiTHAL中已经完成招募的患者给药并持续随访；预计将在2024年年末报告更多的临床数据。自此，由Editas对CRISPR体外项目的开发告一段落，转而面向CRISPR的LNP体内项目。新的重心 Editas推出了未来的重点——针对SCD和β-地贫的LNP体内编辑项目。是的，在放弃已经进入到临床阶段并显示较为积极的数据的SCD和β-地贫项目reni-cel后，Editas瞄准的适应症居然还是SCD和β-地贫，可能是新的LNP体内项目的竞争优势更大吧，毕竟体内LNP是通用型疗法，成本和价格会更低。 Editas利用专有的新型靶向脂质纳米颗粒（t-LNP）开发了下一代SCD和β-地贫体内疗法，并在小鼠模型中完成了临床前概念验证。在人源化小鼠模型（植入人类造血干细胞且缺乏自身造血细胞的小鼠）中，体内疗法实现了造血干细胞和祖细胞（HSPC）的编辑诱导胎儿血红蛋白（HbF）产生。通过利用造血干细胞靶向策略和t-LNP制剂优化，单次给药后，HSPC的编辑水平达到29%。进一步证明，使用t-LNP配方进行的编辑可导致HbF诱导的功能结果，表现为存在表达HbF的人红细胞（平均20%），这些经编辑的红细胞在体内生存一个月。根据Editas报告，截止2024年第三季度，公司拥有约2.65亿美元的现金、现金等价物和有价证券，或在收到DRI的预付款后约为3.2亿美元。体内or体外？摇摆的Editas Editas在业内大名鼎鼎，是张锋创办的第一家公司，是第一家上市的CRISPR基因编辑公司，也是最早将CRISPR技术转化为临床药物的公司。在Editas过去的研发中，其实可以看到其重点在体内或体外疗法的不断摇摆，从AAV体内—到HSPC体外—再到LNP体内。被放弃的AAV体内项目最早先，Editas更多押注在AAV体内项目上，包括其最先推进临床的眼科项目EDIT-101。 EDIT-101是首个获批临床的体内CRISPR基因编辑药物，于2018年获得美国FDA批准IND。但是后来，因为商业潜力堪忧，投入和产出成本不匹配，Editas于2022年宣布为EDIT-101寻求合作机会但无果。延伸阅读：商业潜力堪忧，Editas领先的体内CRISPR疗法停止临床开发 2023年1月，Editas在正式宣布停止对EDIT-101内部投资的同时，又停止了一项AAV体内CRISPR项目EDIT-103。EDIT-103是一款视紫红质相关的常染色体显性视网膜色素变性 (RHO-adRP)疗法，同样临床数据还算积极。另外，被挺停止的还包括一款iNK细胞疗法EDIT-202。转战HSPC体外项目在放弃AAV体内之后，2023-2024年中的Editas将资源集中在HSPC体外项目reni-cel上。经历为时一年半的努力，reni-cel也被放弃。新目标LNP载体在Editas于10月22日发布的向体内CRISPR的战略更新前，Editas于21日宣布与Genevant Sciences达成合作协议。根据协议，双方将Editas的CRISPR Cas12a基因组编辑系统，及Genevant的专有脂质纳米颗粒（LNP）递送技术，共同开发针对特定领域两个未公开靶点的mRNA-CRISPR Cas12a-LNP产品。参考资料： 1.https://ir.editasmedicine.com/news-releases/news-release-details/editas-medicine-announces-progress-towards-2024-goals-including 2.https://ir.editasmedicine.com/news-releases/news-release-details/editas-medicine-and-genevant-sciences-collaborate-develop-novel 3.https://ir.editasmedicine.com/news-releases/news-release-details/editas-medicine-announces-strategic-updates-and-portfolio 本周好文推荐如需转载请联系佰傲谷并在醒目位置注明出处 · · · · · · · ·

基因疗法并购

2024-10-22

·MedCity News

Editas Medicine Seeks Partner for Ex Vivo Gene-Editing Program as Focus Turns to In Vivo R&D - MedCity News

If Editas Medicine’s CRISPR gene-editing therapy for rare blood diseases reaches the market, it will be in the hands of a different company. The biotech on Tuesday announced plans to partner or out-license that clinical-stage ex vivo therapy, choosing instead to focus its resources on in vivo R&D that now has preclinical proof-of-concept data. The in vivo therapy, renizgamglogene autogedtemcel (reni-cel), has reached late-stage clinical development for severe sickle cell disease. Reni-cel is also in early-stage development for transfusion-dependent beta thalassemia. Patients who have these rare blood disorders already have ex vivo gene-editing treatment options. Last winter Vertex Pharmaceuticals’ Casgevy landed FDA approvals for sickle cell disease and beta thalassemia. Bluebird Bio’s last winter received FDA approval for Lygenia for sickle cell disease. A different Bluebird gene-editing therapy named Zynteglo won FDA approval in 2022 for beta thalassemia. Editas’s reni-cel would be third to market, but the Cambridge, Massachusetts-based company has said it could be best in the class of ex vivo therapies, made by editing a patient’s hematopoietic stem cells in a lab and infusing those cells back into the patient. These modified cells are intended to correct the anemia caused by these blood disorders and increase levels of fetal hemoglobin. presented by Sponsored Post The Promise of Value-Based Care and MedTech Innovation Monica Vajani, Executive Director for mHUB’s MedTech Accelerator, discusses how mHUB is helping innovators transition healthcare towards value-based care. By Monica Vajani - mHUB Dosing is continuing in reni-cel’s pivotal sickle cell study with 28 adult patients dosed to date; dosing is being scheduled for adolescent patients. Editas said data will be presented during the upcoming American Society of Hematology meeting in December. The beta thalassemia study is on track to present clinical data by the end of this year. Speaking during a Tuesday webcast, Editas CEO Gilmore O’Neill said the company is mindful of the financial cost that comes with commercializing an ex vivo autologous therapy. Placing reni-cel in the hands of another company would allow Editas to substantially reduce its 2025 spending. “We have previously said that we would be open to partnering reni-cel, but now, with the progress we’re making on in vivo editing, we are actively undertaking a process to partner or out-license reni-cel to most effectively drive it to commercialization while allowing us to apply our full attention and capital to in vivo medicines,” O’Neill said. “The in vivo data we are sharing today makes us even more confident in this decision.” The data that has Editas excited show proof of concept for its in vivo editing approach in mouse models for both sickle cell disease and beta thalassemia. Specifically, the company reported its in vivo therapy enabled an editing level of 29% in hematopoietic stem cells after a single dose. This treatment led to fetal hemoglobin induction (HbF), indicated by the presence of HbF-expressing human red blood cells populating in the host by one month. O’Neill said this level of in vivo editing in mice after a single dose is “highly competitive” compared to data in the public domain for the development of an in vivo medicine for sickle cell disease and beta thalassemia. presented by Health Tech What’s Keeping Healthcare CIOs Up at Night: How to Improve Patient Satisfaction by Handling Calls More Efficiently Health systems have spent billions on portals while investments in modernizing the voice channel — the dominant preference of healthcare consumers — have taken a backseat. By Stephanie Baum In a note sent to investors, Leerink Partners analyst Mani Foroohar said the decision to seek an external partner for reni-cel makes sense given the cost of commercializing autologous ex vivo therapies. The near-term focus for Editas shares is the additional reni-cel data and the extent to which the company can show differentiation for the therapy to drive business development interest in this program. Editas said it has engaged investment bank Moelis & Company to lead the global process to partner or out-license reni-cel. Meanwhile, the company has secured non-dilutive financing to support its in vivo gene-editing focus. Earlier this month, Editas sold to DRI Healthcare Trust certain future license fees and payments owed under a Vertex Pharmaceuticals’ licensing agreement for Cas9 gene-editing technology. The DRI deal paid Editas $57 million up front. Editas reported its cash position as of the end of the third quarter of this year was about $265 million; or about $320 million including the DRI payment. O’Neill said the company has enough capital to fund operations into 2026. Public domain image by Flickr user NIH Image Gallery

引进/卖出基因疗法ASH会议上市批准财报

100 项与 Betibeglogene autotemcel 相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
-	Betibeglogene autotemcel	B06AX	-

研发状态

批准上市

10 条最早获批的记录,

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适应症	国家/地区	公司	日期
地中海贫血	美国	bluebird bio, Inc.	2022-08-17
β地中海贫血	英国	bluebird bio, Inc.	-

未上市

10 条进展最快的记录,

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适应症	最高研发状态	国家/地区	公司	日期
镰状细胞血症	申请上市	美国	bluebird bio, Inc.	2023-04-25
β地中海贫血	申请上市	美国	bluebird bio, Inc.	2022-04-19
输血依赖性地中海贫血	临床3期	英国	bluebird bio, Inc.	2016-08-08
输血依赖性地中海贫血	临床3期	意大利	bluebird bio, Inc.	2016-08-08
输血依赖性地中海贫血	临床3期	德国	bluebird bio, Inc.	2016-08-08
输血依赖性地中海贫血	临床3期	泰国	bluebird bio, Inc.	2016-08-08

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临床结果

适应症

分期

评价

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


HGB-210 (BusinessWire) 人工标引	N/A	镰状细胞血症	47	LYFGENIA	廠積範醖衊構醖膚廠餘(積鑰鏇廠築夢鑰壓繭醖) = 顧繭簾齋網夢築繭鏇壓鹽範夢廠鏇鹹鹽壓膚網 (膚鏇艱簾艱壓憲醖鹹衊 ) 更多	积极	2023-12-09
FDA 人工标引	临床1/2期	镰状细胞血症	45	LYFGENIA	(夢選獵顧選顧窪範獵齋) = 觸積鹹網醖壓築餘積繭鑰憲憲遞窪築鬱蓋範糧 (襯憲鹹鑰淵衊簾範餘窪, 71 ~ 97) 更多	积极	2023-12-08
HGB-206 (HGB-206, Tandem Meetings ASTCT and CIBMTR2022)	临床1/2期	BB305 lentiviral vector (LVV)	-	Lentiglobin (Group A)	淵襯願蓋蓋選觸艱醖窪(壓餘齋齋餘衊簾糧淵網) = The most common SAEs in Groups A and C are shown in Table 2. Two AML events were reported in Group A, and none in Group C. 網觸壓鏇鏇齋齋壓觸艱 (淵齋鏇憲夢壓鹽廠鬱願 )	积极	2022-03-01
HGB-206 (HGB-206, Tandem Meetings ASTCT and CIBMTR2022)	临床1/2期	BB305 lentiviral vector (LVV)	-	Lentiglobin (Group C)		积极	2022-03-01
NCT02906202 (Pubmed \| Br Dent J) 人工标引	临床3期	地中海贫血	23	Betibeglogene autotemcel	(蓋鏇壓繭鑰襯製醖獵齋) = 糧鏇襯鹽鬱簾襯艱鹹積鹹選鹽淵鬱襯鑰衊憲簾 (獵壓蓋餘遞窪網積繭願 ) 更多	积极	2021-12-11
HGB-207, HGB-212 (EHA2021)	临床3期	输血依赖性β地中海贫血	27	betibeglogene autotemcel gene therapy	積夢衊鹹獵齋蓋衊鏇艱(顧壓鏇製廠簾膚網廠鹹) = thrombocytopenia, tachycardia (2 patients < 12 years of age), abdominal pain (2 patients 12-18 years of age) 願構艱壓積獵糧顧鑰夢 (壓鏇製選壓憲範廠網襯 ) 更多	-	2021-06-09
NCT03207009 \| NCT02906202 (Northstar-3, EHA2021)	临床3期	输血依赖性β地中海贫血	41	BETIBEGLOGENE AUTOTEMCEL (Beti-cel)	鑰範鬱遞構蓋醖選醖鏇(餘觸製鬱鏇鑰鬱窪鬱糧) = 範簾範廠壓蓋廠艱艱衊積構襯襯鏇餘齋襯衊襯 (顧夢蓋餘選膚獵艱繭廠 ) 更多	-	2021-06-09
NCT03207009 (Northstar-3, EBMT2021)	临床3期	输血依赖性β地中海贫血 Î²0/Î²+ (IVS-I-110) genotype	1	Betibeglogene Autotemcel (Beti-Cel; Lentiglobin	(襯選鹽衊積壓膚顧觸糧) = grade 2, during infusion, possibly related to treatment 醖窪夢鑰積鏇築顧簾鏇 (遞鑰壓鬱艱醖遞齋鬱衊 ) 更多	积极	2021-03-14
NCT02906202 (EHA2020) 人工标引	临床3期	β地中海贫血	21	Betibeglogene autotemcel	(構齋糧廠積夢壓積構廠) = stomatitis (n=12), febrile neutropenia (n=7), epistaxis (n=3), pyrexia (n=3), and veno-occlusive liver disease (n=3) 積鏇鬱憲獵遞襯觸範獵 (鹽壓齋衊餘齋鏇衊獵觸 ) 更多	积极	2020-05-14
NCT03207009 (EHA2020) 人工标引	临床3期	输血依赖性地中海贫血	13	Betibeglogene autotemcel	襯網艱鬱網鹹願網積觸(齋觸選願願遞簾獵憲壓) = 9/11 pts（≥3 months）築憲鹽範夢淵範廠簾遞 (蓋壓艱蓋膚膚顧壓蓋構 ) 更多	积极	2020-05-14
EHA2020	N/A	β地中海贫血	3	lentiviral vector TNS9.3.55 (TNS9.3.55 vector)	願簾齋繭鏇夢齋窪夢膚(齋壓網廠鏇鹹襯積網繭) = 窪簾製鏇獵鹹範網鏇憲鏇築餘網獵顧壓範艱夢 (鏇顧積選餘衊蓋襯積構 ) 更多	-	2020-05-14